Refractory infantile high-grade glioma containing TRK-fusion responds to
High-grade gliomas (HGGs) are a challenging group of brain tumors.
Identification of targetable genetic alterations such as TRK fusions are
providing new treatment options. We discuss an infant who presented with
hydrocephalus and a cystic mass at birth, while a solid tumor component
of the mass was only appreciated later. Tumor analysis was consistent
with HGG containing a TRK fusion. Despite treatment with conventional
chemotherapy, his tumor progressed in size. He was started on
larotrectinib and experienced a significant decrease in tumor burden.
This case demonstrates that targeted therapy may be effective for a
subset of TRK fusion-positive CNS tumors.